Improving treatments for children with Facioscapulohumeral Muscular Dystrophy (FSHD)
Official title: Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
Funded at University of Kansas Medical Center · NIH-10909643 · Data updated 2026-11-12
This study is looking to speed up the creation of new treatments for children with FSHD by understanding how the disease changes with age and genetics, and it aims to involve more young patients in clinical trials to find better therapies that tackle the root causes of the condition.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Medical Center NIH-funded |
| Lab location | 1 site (Kansas City, United States) |
| Project start | August 20, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2024 |
| Award (this year) | $1,184,364 |
| NIH project number | 1U01NS137702-01 |
| Project ID | NIH-10909643 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with pediatric-onset FSHD, particularly those with early onset and significant disease burden.
✕Less likely to help
Patients with adult-onset FSHD or those without a confirmed diagnosis of FSHD may not benefit from this research.
What this research studies
This research aims to accelerate the development of therapies for pediatric-onset FSHD by validating clinical outcomes and refining strategies for clinical trials. It focuses on understanding how the severity of FSHD varies with age of onset and genetic factors, particularly the D4Z4 repeat number. By utilizing a network of clinical trial sites, the study seeks to recruit and follow a larger number of young patients, which is essential for effective treatment development. The research also explores innovative therapies that target the underlying genetic causes of FSHD, potentially leading to more effective interventions for affected children.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for children with FSHD, improving their quality of life and long-term outcomes.
How similar studies have gone: Previous research has shown promise in developing targeted therapies for FSHD, indicating that this approach could be successful.
Where this research is happening
-
University of Kansas Medical Center
Kansas City, United States Active
Researchers
- Principal investigator: Statland, Jeffrey — University of Kansas Medical Center
- Study coordinator: Statland, Jeffrey
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.